Cost-Effectiveness of Bivalent Respiratory Syncytial Virus Prefusion F (RSVpreF) Vaccine During Pregnancy for Prevention of Respiratory Syncytial Virus Among Infants in Argentina.

INTRODUCTION: Lower respiratory tract illness (LRTI) caused by respiratory syncytial virus (RSV) is common among young children in Argentina. Use of the currently available prophylactic agent is limited to children aged ≤ 2 years with selected high-risk conditions, and thus the majority of infants remain unprotected. We estimated the value-based price (VBP) of a novel RSVpreF vaccine for use among pregnant people for prevention of RSV-LRTI among infants during the first year of life. METHODS: Clinical outcomes and economic costs of RSV-LRTI during infancy and expected impact of RSVpreF vaccination during pregnancy were projected using a population-based Markov-type cohort model. Model results-estimated on the basis of gestational age at birth, disease/fatality rates, and mother's vaccination status-include total numbers of RSV-LRTI cases, RSV-LRTI-related deaths, and associated costs. Base case analyses (RSVpreF vs. no vaccine) were conducted from the healthcare system perspective. Probabilistic sensitivity analyses (PSA; 1000 replications) were also conducted. Willingness-to-pay (WTP) was $10,636 per quality-adjusted life-year (QALY; i.e., 1 × 2021 gross domestic product [GDP] per capita) in base case analyses and PSA. Costs are reported in USD, estimated on the basis of the June 22, 2023 exchange rate. RESULTS: Use of RSVpreF among 342,110 pregnant persons provided protection to 330,079 infants at birth. In total, RSVpreF prevented 3915 RSV hospitalizations, 6399 RSV cases requiring emergency department care, 6182 RSV cases requiring a physician office visit, and 67 disease-related deaths. Direct costs were projected to be reduced by $5.0 million. With 2061 QALYs gained and vaccine administration cost of $1.4 million, the VBP of RSVpreF was estimated to be $74.46 per dose. In PSA, mean VBP was $75.02 (95% confidence interval 54.24-97.30). CONCLUSIONS: RSVpreF among pregnant persons would significantly reduce the clinical and economic burden of RSV-LRTI among infants in Argentina and would be considered a cost-effective intervention up to a price of approximately $75.

Annual public health and economic burden of medically attended respiratory syncytial virus illnesses among US adults.

BACKGROUND: Respiratory syncytial virus (RSV) is a common cause of lower respiratory tract disease (LRTD) among adults and can lead to serious morbidity and mortality; however, evidence on the magnitude of the public health and economic burden of adult RSV-LRTD is limited. This study was undertaken to project annual clinical outcomes and economic costs of medically attended RSV-LRTD among US adults, and to identify subgroups responsible for a disproportionate share of disease burden. METHODS: Clinical outcomes of RSV-LRTD were projected for subgroups of US adults defined by age and comorbidity profile (with vs. without chronic/immunocompromising medical conditions) based on corresponding population sizes, episode (disease) rates, and case-fatality rates. Economic costs comprised medical (i.e., direct) costs and non-medical (i.e., indirect) costs of RSV-LRTD, and were generated based on numbers of episodes and unit costs in relation to setting of care, age, and comorbidity profile. RESULTS: Among 265 million US adults aged ≥18 years in 2023, 6.5 million medically attended episodes of RSV-LRTD were projected to occur including 349,260 requiring hospitalization, 357,892 requiring an emergency department visit (not leading to hospitalization), and 5.8 million requiring other ambulatory care. Direct costs ($15.2 billion) and indirect costs ($9.7 billion) were projected to total $25.0 billion. Persons aged 60-99 years accounted for 31 % of the adult population and over 50 % of the economic burden of RSV-LRTD, while adults aged <60 years with chronic/immunocompromising medical conditions accounted for 10 % of the population and 27 % of the economic burden. CONCLUSIONS: Annual burden of RSV-LRTD among US adults-especially older adults and those of all ages with underlying medical conditions-is substantial. Preventive measures, such as recently approved RSV vaccines, have the potential to yield important improvements in public and patient health, and to reduce the economic burden of RSV-LRTD from the US healthcare system and societal perspectives.

Modified blood cell GAP model as a prognostic biomarker in idiopathic pulmonary fibrosis.

Background: The Gender, Age and Physiology (GAP) model is a simple mortality prediction tool in patients with idiopathic pulmonary fibrosis that uses demographic and physiological variables available at initial evaluation. White blood cell variables may have associations with idiopathic pulmonary fibrosis outcomes. We evaluated whether incorporating blood cell counts in modified GAP (cGAP) models would improve outcome prediction in patients with idiopathic pulmonary fibrosis. Patients and methods: This retrospective analysis included pooled data from phase 3 randomised trials of pirfenidone in idiopathic pulmonary fibrosis (ASCEND, CAPACITY 004, CAPACITY 006). Study outcomes (disease progression, all-cause mortality, all-cause hospitalisation, respiratory-related hospitalisation) were evaluated during the initial 1-year period. Shared frailty models were used to evaluate associations between continuous and categorical baseline white and red blood cell parameters and study outcomes in a bivariate context, and to evaluate the impact of adding continuous monocyte count (cGAP1) or white and red blood cell parameters (cGAP2) to traditional GAP variables in a multivariable context based on C-statistics changes. Results: Data were pooled from 1247 patients (pirfenidone, n=623; placebo, n=624). Significant associations (bivariate analyses) were idiopathic pulmonary fibrosis progression with neutrophil and eosinophil counts; all-cause mortality with monocyte and neutrophil counts; all-cause hospitalisation with monocyte count, neutrophil count and haemoglobin level; and respiratory-related hospitalisation with monocyte count, neutrophil count and haemoglobin level. In multivariate analyses, C-statistics were highest for the cGAP2 model for each of the outcomes. Conclusion: Modified GAP models incorporating monocyte counts alone or plus other white and red blood cell variables may be useful to improve prediction of outcomes in patients with idiopathic pulmonary fibrosis.

Cost-Effectiveness of 20-Valent Pneumococcal Conjugate Vaccine Among US Children with Underlying Medical Conditions.

INTRODUCTION: A 20-valent pneumococcal conjugate vaccine (PCV20) was recently recommended for use among US children. We evaluated the cost-effectiveness of PCV20 among children aged 6 years with chronic medical conditions (CMC+) and children aged 6 years with immunocompromising conditions (IC) versus one and two doses of 23-valent pneumococcal polysaccharide vaccine (PPSV23), respectively. METHODS: A probabilistic model was employed to depict 10-year risk of clinical outcomes and economic costs of pneumococcal disease, reduction in life years from premature death, and expected impact of vaccination among one cohort of children with CMC+ and IC aged 6 years. Vaccine uptake was assumed to be 20% for both PCV20 and PPSV23. Cost per quality-adjusted life year (QALY) gained was evaluated from the US societal and healthcare system perspectives; deterministic and probabilistic sensitivity analyses (DSA/PSA) were also conducted. RESULTS: Among the 226,817 children with CMC+ aged 6 years in the US, use of PCV20 (in lieu of PPSV23) was projected to reduce the number cases of pneumococcal disease by 5203 cases, medical costs by US$8.7 million, and nonmedical costs by US$6.2 million. PCV20 was the dominant strategy versus PPSV23 from both the healthcare and societal perspectives. In the PSA, 99.9% of the 1000 simulations yielded a finding of dominance for PCV20. Findings in analyses of children with IC aged 6 years in the USA were comparable (i.e., PCV20 was the dominant vaccination strategy). Scenario analyses showed that increasing PCV20 uptake to 100% could potentially prevent > 22,000 additional cases of pneumococcal disease and further reduce medical and nonmedical costs by US$70.0 million among children with CMC+ and IC. CONCLUSIONS: Use of PCV20 among young children with CMC+ and IC in the USA would reduce the clinical burden of pneumococcal disease and yield overall cost savings from both the US healthcare system and societal perspectives. Higher PCV20 uptake could further reduce the number of pneumococcal disease cases in this population.

Attributable Cost of Adult Respiratory Syncytial Virus Illness Beyond the Acute Phase.

Background: Estimates of the cost of medically attended lower respiratory tract illness (LRTI) due to respiratory syncytial virus (RSV) in adults, especially beyond the acute phase, is limited. This study was undertaken to estimate the attributable costs of RSV-LRTI among US adults during, and up to 1 year after, the acute phase of illness. Methods: A retrospective observational matched-cohort design and a US healthcare claims repository (2016-2019) were employed. The study population comprised adults aged ≥18 years with RSV-LRTI requiring hospitalization (RSV-H), an emergency department visit (RSV-ED), or physician office/hospital outpatient visit (RSV-PO/HO), as well as matched comparison patients. All-cause healthcare expenditures were tallied during the acute phase of illness (RSV-H: from admission through 30 days postdischarge; ambulatory RSV: during the episode) and long-term phase (end of acute phase to end of following 1-year period). Results: The study population included 4526 matched pairs of RSV-LRTI and comparison patients (RSV-H: n = 970; RSV-ED: n = 590; RSV-PO/HO: n = 2966). Mean acute-phase expenditures were $42 179 for RSV-H (vs $5154 for comparison patients), $4409 for RSV-ED (vs $377), and $922 for RSV-PO/HO (vs $201). By the end of the 1-year follow-up period, mean expenditures-including acute and long-term phases-were $101 532 for RSV-H (vs $36 302), $48 701 for RSV-ED (vs $27 131), and $28 851 for RSV-PO/HO (vs $20 523); overall RSV-LRTI attributable expenditures thus totaled $65 230, $21 570, and $8327, respectively. Conclusions: The cost of RSV-LRTI requiring hospitalization or ambulatory care among US adults is substantial, and the economic impact of RSV-LTRI may extend well beyond the acute phase of illness.

Rates of Lower Respiratory Tract Illness in US Adults by Age and Comorbidity Profile.

INTRODUCTION: While it is widely recognized that older adults, adults with chronic medical conditions (CMC), and adults with immunocompromising conditions (IC) are at increased risk of lower respiratory tract illness (LRTI), evidence of the magnitude of increased risk is limited. This study was thus undertaken to characterize rates of hospitalized and ambulatory LRTI among United States (US) adults by age and comorbidity profile. METHODS: A retrospective cohort design and US healthcare claims database (2016-2019) were employed. Study population included adults aged ≥ 18 years and was stratified by age and comorbidity profile (CMC-, CMC+ , IC). LRTI was ascertained overall and by pathogen pathogen (e.g., respiratory syncytial virus [RSV]), and was classified by care setting (hospital, emergency department [ED], physician office/hospital outpatient [PO/HO]). RESULTS: Relative rates (RR) of LRTI generally increased with older age across care settings (vs. 18-49 years), with the most marked increase for hospitalizations: for LRTI-hospitalized, RRs ranged from 3.3 for 50-64 years to 46.6 for ≥ 85 years; for LRTI-ED and LRTI-PO/HO, RRs ranged from 1.0 to 2.7 and from 1.3 to 1.5, respectively. Within age groups, LRTI rates were also consistently higher among CMC+ and IC adults (vs. CMC- adults). Age-specific RRs of LRTI patients hospitalized due to RSV were largely comparable to overall LRTI; age-specific RRs for other care settings, and RRs for CMC+ and IC adults (vs. CMC- adults), were generally higher for LRTI due to RSV. CONCLUSIONS: Incidence of LRTI, including that due to RSV, especially for events requiring acute inpatient care, is markedly higher among older adults and adults of all ages with CMC or IC.

A formula for predicting emphysema extent in combined idiopathic pulmonary fibrosis and emphysema.

BACKGROUND: No single pulmonary function test captures the functional effect of emphysema in idiopathic pulmonary fibrosis (IPF). Without experienced radiologists, other methods are needed to determine emphysema extent. Here, we report the development and validation of a formula to predict emphysema extent in patients with IPF and emphysema. METHODS: The development cohort included 76 patients with combined IPF and emphysema at the Royal Brompton Hospital, London, United Kingdom. The formula was derived using stepwise regression to generate the weighted combination of pulmonary function data that fitted best with emphysema extent on high-resolution computed tomography. Test cohorts included patients from two clinical trials (n = 455 [n = 174 with emphysema]; NCT00047645, NCT00075998) and a real-world cohort from the Royal Brompton Hospital (n = 191 [n = 110 with emphysema]). The formula is only applicable for patients with IPF and concomitant emphysema and accordingly was not used to detect the presence or absence of emphysema. RESULTS: The formula was: predicted emphysema extent = 12.67 + (0.92 x percent predicted forced vital capacity) - (0.65 x percent predicted forced expiratory volume in 1 second) - (0.52 x percent predicted carbon monoxide diffusing capacity). A significant relationship between the formula and observed emphysema extent was found in both cohorts (R2 = 0.25, P < 0.0001; R2 = 0.47, P < 0.0001, respectively). In both, the formula better predicted observed emphysema extent versus individual pulmonary function tests. A 15% emphysema extent threshold, calculated using the formula, identified a significant difference in absolute changes from baseline in forced vital capacity at Week 48 in patients with baseline-predicted emphysema extent < 15% versus ≥ 15% (P = 0.0105). CONCLUSION: The formula, designed for use in patients with IPF and emphysema, demonstrated enhanced ability to predict emphysema extent versus individual pulmonary function tests. TRIAL REGISTRATION: NCT00047645; NCT00075998.

The critical role of phlebolymphedema in cellulitis associated with lymphedema: Its incidence and economic impact in a large real-world population.

OBJECTIVE: The aims of this study were: to define the incidence of cellulitis in patients with lymphedema (LED) overall and relate this to the etiology of LED; to determine how this rate might be affected by recurrence of cellulitis; and to quantify the contemporary economic burden of treatment. Understanding these factors is essential in developing targeted cellulitis prevention strategies and reducing health care costs. METHODS: The IBM MarketScan Research Database was examined from April 2013 to March 2019 for patients with a new diagnosis of LED (n = 85,601). Based on International Classification of Diseases (ICD)-9/ICD-10 diagnosis codes, the incidence and cost of cellulitis were ascertained during the 3-year follow-up period. Incidence rates (per 100 patient-years [PYs]) and cost (per patient per year) of cellulitis were evaluated among all patients with LED and within subgroups of LED etiologies. RESULTS: Among the three most common morbidities associated with LED (breast cancer-related lymphedema [BCRL], n = 17,954 [20.97%]; gynecological cancer-related LED [GCRL], n = 1256 [1.47%]; and phlebolymphedema [PLED], n = 8406 [9.82%]), rates of cellulitis were markedly lower for BCRL (8.9; 95% confidence interval [CI], 8.7-9.2) and GCRL (14.8; 95% CI, 13.4-16.4) vs PLED (47.7; 95% CI, 46.7-48.8). Patients with a history of cellulitis had markedly higher cellulitis rates during follow-up than those without-overall, 74.0% vs 16.4%; BCRL, 42.9%; 95% CI, 39.7%-46.3% vs 7.6%; 95% CI, 7.3%-7.9%; GCRL, 67.5%; 95% CI, 56.4%-80.8% vs 11.0%; 95% CI, 9.8%-12.4%; and PLED, 81.7%; 95% CI, 79.4%-84.1% vs 30.4%; 95% CI, 29.4%-31.4%, respectively. The mean $/patient/year of cellulitis-related costs for a patient with PLED ($2836; 95% CI, $2395-$3471) was significantly greater than that for BCRL ($503; 95% CI, $212-$1387) and GCRL ($609; 95% CI, $244-$1314). CONCLUSIONS: The incidence of cellulitis associated with LED varies by the etiology of LED. PLED has the highest rates of both an initial cellulitis episode and recurrent cellulitis events. Additionally, PLED has one of the largest cellulitis-related total costs per patient per year. Prevention, as well as early identification and treatment of PLED-associated cellulitis, could significantly decrease health care costs and improve patient quality of life.

Lymphoscintigraphy is frequently recommended but seldom used in a "real world setting".

OBJECTIVE: Lymphedema (LED) lacks a standard, simple, guiding noninvasive diagnostic test, unlike the two other circulatory disorders-arterial or venous disease. Lymphoscintigraphy (LSG) has been recommended by several guidelines as the diagnostic test of choice for LED. Several recent expert panels, however, have suggested from anecdotal experience that LSG was used infrequently, and that the diagnosis of LED is usually based on clinical examination. METHODS: To determine the use of LSG in a large real-world LED population, the International Business Machines MarketScan Research Database was examined from April 2012 to March 2020 for patients with a new diagnosis of LED (the index date). Use of LSG (LSG+) was ascertained during the period beginning 12 months prior to the initial coding of a LED diagnosis and ending 12 months after the index date based on the corresponding Current Procedural Terminology code; LSG use for sentinel node mapping at the time of oncologic surgery was excluded. Demographic profiles, comorbidities, and causes of LED among patients with and without evidence of LSG were characterized. RESULTS: We identified 57,674 patients, aged ≥18 years, who had a new diagnosis of LED and health care coverage for ≥12 months before and after this index date. Only a small number (1429; 2.5%) of these patients underwent LSG during the study period. The LSG + cohort was younger (53.7 vs 60.7 years), had a higher proportion of women (91.3% vs 73.4%), but a lower percentage of diabetes (12.8% vs 27.5%), heart failure (2.2% vs 8.7%), hypertension (32.4% vs 51.0%), and obesity (15.1% vs 22.2%) compared with the LED population who did not undergo LSG (all P < .001). Most importantly, the use of LSG for diagnosis varied with the etiology of LED (LSG was most frequently utilized among patients with melanoma-LED (9.5%) and patients with breast cancer-LED (6.7%), in contrast to patients with advanced venous disease-related LED (1.1%; P < .05 for both comparisons). CONCLUSIONS: Despite four guidelines recommending LSG, including the Guidelines of the American Venous Forum (Handbook of Venous and Lymphatic Disease-4th edition), which recommended LSG "for the initial evaluation of patients with LED" with a 1B recommendation, LSG plays a minor role in establishing the diagnosis of LED in the United States. This underlines the need for a better, simple diagnostic test for LED to complement clinical examination.

Episodic Cost of Lower Respiratory Tract Illness due to Respiratory Syncytial Virus Among US Infants During the First Year of Life.

A study of 2 health care claims databases (commercial, Medicaid) was undertaken to estimate the episodic cost of lower respiratory tract illness due to respiratory syncytial virus among infants aged <12 months overall, by age, and by birth gestational age. Among commercial-insured infants, mean costs were $28 812 for hospitalized episodes, $2575 for emergency department episodes, and $336 for outpatient clinic episodes. Costs were highest among infants aged <1 month and infants with a gestational age ≤32 weeks and were comparable among Medicaid-insured infants, albeit somewhat lower. The cost of lower respiratory tract illness due to respiratory syncytial virus during the acute phase of illness is high, especially among the youngest infants and those born premature.

Impact of Elexacaftor/Tezacaftor/Ivacaftor on Healthcare Resource Utilization and Associated Costs Among People With Cystic Fibrosis in the US: A Retrospective Claims Analysis.

INTRODUCTION: Cystic fibrosis (CF) is a life-limiting genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) is a CFTR modulator (CFTRm) that targets the underlying cause of CF. Based on safety and efficacy demonstrated in clinical trials, ELX/TEZ/IVA is approved in the US for the treatment of CF in people aged ≥ 2 years who have ≥ 1 F508del-CFTR mutation or a CFTR mutation that is responsive to ELX/TEZ/IVA based on in vitro data. While ELX/TEZ/IVA demonstrated unprecedented improvements in lung function and dramatic reductions in pulmonary exacerbations (PEx) and associated hospitalizations in clinical trials, a limited number of studies have examined the impact of ELX/TEZ/IVA on healthcare resource utilization (HCRU) and associated costs in a real-world setting. The aim of this retrospective study was to evaluate changes in PEx, HCRU, and associated non-CFTRm healthcare costs following initiation of ELX/TEZ/IVA among people with CF aged ≥ 12 years in the US. METHODS: We evaluated the rates of PEx, HCRU, and associated costs before and after initiation of ELX/TEZ/IVA in people with CF aged ≥ 12 years using data from the Merative MarketScan® Commercial Claims and Encounters Database and the Merative Multi-State Medicaid Database from April 21, 2019 to December 31, 2020. Because the study period included time following the onset of the COVID-19 pandemic, we limited our primary analysis to the period prior to the pandemic (October 21, 2019 to March 12, 2020). Outcomes following the onset of the pandemic (March 13 to December 31, 2020) were examined in an exploratory analysis. RESULTS: In both commercially insured and Medicaid-insured people with CF, ELX/TEZ/IVA was associated with reductions in PEx, hospitalizations, and associated costs prior to the COVID-19 pandemic, and these reductions were maintained following the onset of the pandemic. CONCLUSIONS: These findings suggest that ELX/TEZ/IVA reduces the burden and costs associated with PEx and hospitalizations in people with CF.

Impact of lymphedema in the management of venous leg ulcers.

INTRODUCTION: Lymphedema (LED) in patients with venous leg ulcers (VLU) [VLU+LED] can impair ulcer healing and predispose to cellulitis. There is little data, however, demonstrating how lymphatic dysfunction may impact the clinical course, treatment, and healthcare expenditures for VLU+LED versus VLU-LED patients. OBJECTIVE: To determine how lymphatic dysfunction might influence treatment and expenditures among VLU patients in a large deidentified healthcare claims database. METHODS: A retrospective cohort design and data from the IBM MarketScan Database (April 2013 to March 2019) were employed. Study population comprised VLU patients, and was stratified into two subgroups: VLU+LED (index date = date of first LED diagnosis) and VLU-LED (index dates randomly assigned to match distribution of index dates for VLU+LED). Within each subgroup, patients with <1 year of healthcare claims information before and after their index dates were excluded. Demographics, comorbidities, procedures/treatments, as well as all-cause post-index medical resource utilization and expenditures ($/patient/year) of the two groups were compared. Stabilized inverse probability treatment weights (IPTWs) were employed to adjust for differences between groups in baseline characteristics. RESULTS: A total of 5466 VLU patients were identified (VLU+LED: N = 299; VLU-LED: N = 5167). Overall ambulatory encounters (AMB ENC) and their components were higher in VLU+LED, which were reflected in increased expenditures for this group (Table 1). Treatment with endovenous ablation (EVA) or stenting for venous hypertension as well as for specific measures for LED were higher in the 1-year post-index period for VLU+LED. The use of LED specific therapy was low for both groups, but a greater percentage of VLU+LED patients received therapy, which was predominantly manual lymphatic drainage (17.4%) rather than pneumatic compression (10.7%). CONCLUSIONS: The clinical presence of LED in patients with VLU is a marker for a more complex disease process with more episodes of cellulitis and expenditures, but a surprisingly low specific treatment for LED.

Pneumococcal vaccine uptake among Medicare Beneficiaries aged ≥65 years following the shared clinical decision-making recommendation for 13-valent pneumococcal conjugate vaccine in 2019.

BACKGROUND: In November 2019, the US Advisory Committee on Immunization Practices recommended shared clinical decision-making (SCDM) for use of 13-valent pneumococcal conjugate vaccine (PCV13) among immunocompetent elderly adults. The impact of SCDM on PCV13 use in this population, immunocompromised persons, and vulnerable subgroups has not been well documented. METHODS: Using Medicare Research Identifiable Files (01/2018 - 09/2020), monthly uptake of pneumococcal vaccine (PCV13, 23-valent pneumococcal polysaccharide vaccine [PPSV23]) was identified among fee-for-service beneficiaries aged ≥ 65 years with Part B coverage and no evidence of prior PCV13. Uptake was stratified by vaccine, risk profile, and demographics. RESULTS: Among the > 12 M beneficiaries included each month, PCV13 uptake declined from > 70% of pneumococcal vaccinations before SCDM to < 60% after SCDM (02/2020). Reductions in PCV13 uptake were consistent across vulnerable subgroups as well as immunocompromised persons. CONCLUSIONS: PCV13 use decreased among immunocompetent and immunocompromised persons alike, despite continued routine PCV13 recommendation for the latter group.

Hemoglobin and End-Organ Damage in Individuals with Sickle Cell Disease.

Background: Sickle cell disease (SCD) is an inherited, chronic, multifaceted blood disorder. Patients with SCD develop anemia, which has been associated with end-organ damage (EOD). Objectives: This retrospective, observational, repeated-measures study systematically characterizes the relationship between hemoglobin (Hb) level and EOD in adolescent and adult patients with SCD. Methods: The study population comprised patients with SCD aged ≥12 years with available Hb data from a US provider-centric health care database. For each patient, each Hb value over time was included as a separate observation. Study outcomes-the onset of any new EOD, including chronic kidney disease, pulmonary hypertension, stroke, and leg ulcer-were ascertained during the 1-year period after each Hb assessment. The association between Hb levels and risk of new EOD was estimated using multivariable generalized estimating equations. Results: A total of 16,043 unique patients with SCD contributed 44,913 observations. Adjusted odds of any EOD during the 1-year follow-up were significantly lower with higher Hb level. Risk reductions with higher Hb levels for chronic kidney disease, pulmonary hypertension, and leg ulcer were comparable. The risk of new EOD was significantly lower among adolescent and adult patients with higher Hb levels. Conclusions: In patients with SCD, higher Hb levels are associated with a reduced risk of developing EOD. Therapeutic strategies that result in higher Hb levels may offer clinical and economic value for patients with SCD. (Curr Ther Res Clin Exp. 2023; 84:XXX-XXX).

Under recognition and treatment of lymphedema in head and neck cancer survivors - a database study.

PURPOSE: Head and neck cancer (HNC) will be diagnosed in approximately 54,000 Americans in 2022 with more than 11,000 dying as a result. The treatment of HNC often involves aggressive multimodal therapy including surgery, radiotherapy, and systemic therapy. HNC and its treatments are associated with multiple painful and function-limiting neuromusculoskeletal and visceral long-term and late effects. Among these is head and neck lymphedema (HNL), the abnormal accumulation of protein rich fluid, in as many as 90% of survivors. Though HNL is common and potentially contributory to other function-limiting issues in this population, it is notoriously understudied, underrecognized, underdiagnosed, and undertreated. This study seeks to determine the incidence of HNC-related lymphedema diagnosis and treatment in a large US healthcare claims repository database. METHODS: A retrospective observational cohort design and data from an integrated US healthcare claims repository-the IBM MarketScan Commercial Claims and Encounters (CCAE) and Medicare Supplemental and Coordination of Benefits (MDCR) Databases spanning the period April 1, 2012 through March 31, 2020. RESULTS: Of the 16,654 HNC patients eligible for evaluation, 1,082 (6.5%) with a diagnosis of lymphedema were identified based on eligibility criteria. Of the 521 HNC patients evaluated for lymphedema treatment, 417 (80.0%) patients received 1.5 courses of MLD, 71 (13.6%) patients were prescribed compression garments, and 45 (8.6%) patients received an advanced pneumatic compression device. CONCLUSION: HNL in this population of HNC survivors was underdiagnosed and treated compared with contemporary assessments HNL incidence.

Clinical Impact of Severe Obesity in Patients with Lymphoedema.

OBJECTIVE: With the rate of obesity increasing worldwide, patients with lymphoedema with and without a concomitant diagnosis of severe obesity (SO) were compared in regard to their baseline demographics, health related characteristics, treatment plans, and patient outcomes. METHODS: This was a retrospective observational cohort study. The IBM MarketScan database was examined (2013 - 2019) for patients with a new diagnosis of lymphoedema. Of 60 284 patients with lymphoedema identified, 6 588 had SO defined by a body mass index > 40 kg/m2. The demographics and other characteristics of SO were compared with patients with lymphoedema without SO. RESULTS: SO and lymphoedema diagnosis increased two fold from 2013 to 2019. The lymphoedema SO+ group was younger (57.8 vs. 60.8 years, p < .001) and with a higher proportion of men (37.7% vs. 24.9%, p < .001) than the lymphoedema SO- group. More comorbidities were observed in the lymphoedema SO+ group than the lymphoedema SO- group: diabetes 46.0% vs. 24.9 % (p < .001), heart failure 18.3% vs. 7.4% (p < .001), hypertension 75.0% vs. 47.6% (p < .001), and renal disease 24.8% vs. 11.9% (p < .001). Use of diuretics in the lymphoedema SO+ group was greater: 57.6% vs. 38.0% (p < .001). Patients with lymphoedema SO+ had higher risk of cellulitis: 34.5% vs. 13.5% (p < .001). Specific lymphoedema treatment was given more often to lymphoedema SO-: 66.3% vs. 64.3% (p = .003). This was significant for manual lymphatic drainage (46.6% vs. 40.0%; p < .001) and physical therapy (55.4% vs. 51.6%; p<.001), but not for compression garments (18.2% vs. 17.7%; p = .38). However, more patients with lymphoedema SO+ received pneumatic compression device treatment: 20.9% vs. 13.7% (p < .001). CONCLUSION: There was an increase in SO associated lymphoedema. Patients with lymphoedema SO+ have over a two and half fold increase in cellulitis incidence, with a significant increase in medical resource use and cost. Despite this, patients with lymphoedema and SO receive less specific therapy such as compression, which has proven to reduce cellulitis incidence.

Cost-effectiveness of using a 20-valent pneumococcal conjugate vaccine to directly protect adults in England at elevated risk of pneumococcal disease.

OBJECTIVES: Despite the current pneumococcal vaccination program in England for older adults and adults with underlying conditions, disease burden remains high. We evaluated cost-effectiveness of 20-valent pneumococcal conjugate vaccine (PCV20) compared to current pneumococcal recommendations for adults in England. METHODS: Lifetime outcomes/costs of invasive pneumococcal disease (IPD) and community-acquired pneumonia (CAP) among adults aged 65-99 years and adults aged 18-64 years with underlying conditions in England were projected using a deterministic cohort model. Vaccination with PCV20 was compared with 23-valent pneumococcal polysaccharide vaccine (PPV23) from the National Health Service perspective. RESULTS: PCV20 was cost saving compared with PPV23 in base case and most sensitivity analyses. In the base case, replacing PPV23 with PCV20 prevented 7,789 and 140,046 cases of IPD and hospitalized CAP, respectively, and 22,199 associated deaths, resulting in incremental gain of 91,375 quality-adjusted life-years (QALYs) and incremental savings of £160M. In probabilistic sensitivity analyses, PCV20 (vs. PPV23) was cost saving in 85% of simulations; incremental cost per QALY was below £30,000 in 99% of simulations. CONCLUSIONS: PCV20 vaccination in adults aged 65-99 years and those aged 18-64 years with underlying comorbidities in England is expected to prevent more hospitalizations, save more lives, and yield lower overall costs than current recommendations for PPV23.

Public health and budgetary impact of 20-valent pneumococcal conjugate vaccine for adults in England.

BACKGROUND: Despite use of 23-valent pneumococcal polysaccharide vaccine (PPV23) in England, disease burden among at-risk adults remains high. We evaluated the public health and budgetary impact of 20-valent pneumococcal conjugate vaccine (PCV20) compared to the current adult pneumococcal vaccination program. METHODS: Five-year outcomes and costs of invasive pneumococcal disease (IPD) and community-acquired pneumonia (CAP) among adults aged 65-99 years and adults aged 18-64 years with underlying conditions in England were projected using a deterministic cohort model. Hypothetical vaccination with PCV20 versus PPV23 was compared from the National Health Service (NHS) perspective. RESULTS: Replacing PPV23 with PCV20 would prevent 785 IPD hospitalizations, 11,751 CAP hospitalizations, and 1,414 deaths over 5 years, and would reduce medical care costs by £48.5 M. With vaccination costs higher by £107.2 M, projected net budgetary impact is £58.7 M. The budgetary impact would be greatest in year 1 (£26.3 M), and would decrease over time (to £1.6 M by year 5). The average budget increase (£11.7 M/year) represents <0.01% of the Department of Health and Social Care total budget and <3% of the vaccine budget. CONCLUSIONS: Use of PCV20 among adults currently eligible for PPV23 in England would substantially reduce the burden of pneumococcal disease, with modest budgetary impact.

Real-world effectiveness of voxelotor for treating sickle cell disease in the US: a large claims data analysis.

BACKGROUND: Sickle cell disease (SCD) is a genetic disease that impacts patients' quality of life, healthcare costs, and life expectancy. Elevated sickle hemoglobin (HbS), which readily polymerizes, causes red blood cell sickling, leading to chronic hemolytic anemia and complications often requiring hospitalization and transfusions. In 2019, voxelotor, which inhibits HbS polymerization, was approved for SCD treatment. OBJECTIVES: This study uses real-world evidence to assess voxelotor's effectiveness in SCD patients in typical clinical practice from 2019 to 2021 using a national medical claims database (N = 3128). RESULTS: After initiating voxelotor, 60.8% of patients with available hemoglobin (Hb) laboratory data (n = 74) showed a Hb increase >1 g/dL. Mean transfusion rate per patient-year dropped 52% in patients with ≥1 transfusion before treatment (n = 190). In patients with ≥1 of the corresponding events (n = 1065), decreases were observed in mean vaso-occlusive crisis (VOC) frequency (-23%); mean VOC-related hospitalizations and length of stay (LOS) time (-34% and -30%, respectively); mean all-cause hospitalization and LOS time (-37% and -23%, respectively); outpatient visits (-10%); iron chelation use (-46%); and prescribed opioids (-13%). CONCLUSION: These data align with randomized controlled trial results showing voxelotor improvements and support that voxelotor may lower transfusion and VOC rates in clinical practice.